April 15, 2017 (Vol. 37, No. 8)

Industry Watch: Advisory Panels’ Rebuff Could Have Been Worse for Endo

After a year where shares plunged 74%, the CEO resigned, and his successor began layoffs, Endo International seemed headed for a much calmer 2017 in February when it reported fourth-quarter 2016 results that beat Wall Street forecasts.

Instead, Endo encountered more rough sailing March 14, when a pair of FDA advisory panels concluded that the benefits of the company’s abuse-deterrent extended-release formulation of Opana® ER (oxymorphone) no longer outweighed its risks.

Not surprisingly, Endo disagreed: “The body of evidence established through clinical research demonstrates that Opana ER has a favorable risk-benefit profile when used as intended in appropriate patients,” stated Matthew W. Davis, M.D., senior vice president, R&D, Branded Pharmaceuticals.

The FDA drug safety and risk management and anesthetic and analgesic drug products advisory committees issued their negative risk-benefit assessments of the reformulated Opana in an 18–8 vote. The FDA is not bound by the vote, though the agency typically aligns its decisions with the advice of advisory committees.

While the vote could lead the FDA to actions that include withdrawing Opana from the market, one analyst said Endo faces no immediate threat.

“We expect it could be several months (or longer) before any potential action is taken to pull the reformulated Opana ER from the market,” Canacord Genuity pharmaceuticals analyst Dewey Steadman said. “We believe any potential impact to Endo will be muted, given the product’s small contribution to Endo’s top and bottom lines.”

Opana ER generated $158.938 million in net revenues last year, down 10% from $175.772 million in 2015 thanks to more generic competition. Endo expects revenues from Opana ER and other legacy branded pain treatments to decline this year, as they did in 2016 (down 24% to $486.368 million). Generics revenues is also expected to fall.

Those projected declines, plus divestitures and product discontinuations, explain why Endo is forecasting lower revenues for 2017—between $3.45 billion to $3.60 billion, compared with $4.010 billion in 2016, up 23% from 2015. Despite that jump, Endo finished 2016 with a $3.347 billion loss, more than double 2015’s $1.495 billion loss—and a stock that plummeted from $62.42 to $16.47.

Discovery & Development: Rarefied Cannabinoid Process Achieves New (Biosynthetic) Highs

Although the Trump administration has been giving mixed messages about marijuana legalization, it seems to regard recreational use of marijuana as being relatively base (and worthy of prohibition), and medical use as being relatively elevated (and worthy of forbearance). With time, the administration may define intermediate altitudes. For example, it may place marijuana-containing nutriceuticals below highly processed pharmaceutical-grade products, which may contain marijuana extracts. An even loftier status may be accorded artificial products.

If official favor is to be accorded in proportion to one’s distance from the cannabis plant, a company called Teewinot Life Sciences will be well positioned. Instead of refining and modifying cannabinoids extracted from cannabis, Teewinot manufactures authentic cannabinoids that have the same chemical structure as naturally occurring phytocannabinoids. The company relies on methods that combine the tools of synthetic biology, biocatalysis, and chemical synthesis.

Teewinot recently signed a letter of intent with Noramco, a producer of controlled-substance active pharmaceutical ingredients. Together, the companies plan to enter the commercial cannabinoids market using Teewinot’s CannSynthesis™ biocatalytic technology. Teewinot will also provide the expertise Noramco requires to implement the technology rapidly.

Noramco will create 10–15 cannabinoid reference standards with CannSynthesis technology and simultaneously evaluate it for the production of dronabinol on a commercial scale. Dronabinol is known for its effectiveness in pain management, as an antiemetic, in the control of vomiting, as an appetite stimulant in the treatment of AIDS, and for reducing the side effects of chemotherapy.

“Combining Teewinot’s CannSynthesis™ technology with Noramco’s controlled substance manufacture and distribution expertise allows us to serve a fast-emerging market space,” said James Mish, president and CEO of Noramco.

Projections of the size of the cannabis biotech/pharma market may need to be recalculated, depending on the Trump administration’s actions. If estimates cited in 2015 by Viridian Capital Advisors remain valid, however, the market could surpass $20 billion by 2020. 

Genomics & Proteomics: Omica Rebrands to Fabric Genomics, Signs Key Partnership with Sentieon

 The Oakland, California-based company Omica, a leading provider of clinical genome interpretation software and services, recently revealed its new name, logo, and brand identity, calling itself Fabric Genomics. Concomitantly, the organization launched Fabric EnterpriseTM, an optimized computational genomics platform enabling secondary analysis, rapid annotation, guideline-driven variant classification, and clinical reporting for both hereditary disease and oncology.

“As a result of our recent acquisition, related capability expansions, and technology partnerships, it was time to reimagine our company’s broader role in the future of healthcare,” said Matt Tindall, CEO of Fabric Genomics. “Our role, along with our partners, is to secure, manage, analyze, interpret, and weave genetic data into the fabric of healthcare to drive behavior change, cost efficiency, and better outcomes. The idea that we can unlock genetic information, unlike any other company, to inform important healthcare decisions, gave rise to Fabric Genomics.”

In the midst of its new branding and platform launch, Fabric Genomics also announced a technology partnership with Sentieon, which develops highly optimized algorithms for bioinformatics applications. This new partnership, set to enhance Fabric Genomics’ secondary analysis capabilities within Fabric Enterprise, spawning Fabric StandardTM, which is ten times faster than commonly adopted bioinformatic tools. Fabric Standard has no down-sampling of reads and is deterministic, even when running on multiple threads. This leads to more accurate scientific results and better overall efficiency.

“By adding Sentieon’s award-winning algorithms to Fabric Enterprise, we can deliver a seamless end-to-end solution for genomic data analysis,” Tindall noted. “This marks a broad expansion in our capabilities as a company and provides our customers with the option to work with one software partner for all their NGS testing needs.”

Jun Ye, Ph.D., Sentieon’s CEO added that “working with Fabric Genomics, Sentieon can help to improve patient care around the world with a total solution for clinical care. We anticipate opportunities to build important and powerful new applications with the Fabric Genomics team.”

Bioprocessing: Horizon Discovery and Amplycell Ink Deal for Bioproduction Cell-Line Optimization

Horizon Discovery Group entered into a research and development collaboration and license agreement with Amplycell  to evaluate the impact of Amplycell’s BOOST cell-line optimization technology on Horizon’s parental GS (glutamine synthetase) null CHO-K1 cell line.

Amplycell’s Cell Fitness Technologies, which include BOOST, consist of a four-stage process to optimize cell lines in terms of stability and productivity. Cell lines can be stimulated using AmplyCell’s Cell Fitness Technologies at any stage of cell-line development or bioproduction (clone creation, RCB/DCB, MCB), without the need to change culture conditions, according to Geoffrey Holsbeek, CEO of Amplycell.

Horizon is applying a disruptive business model alongside its expertise in genome editing to improve the manufacture of biotherapeutics, making its CHO cells available to companies of all sizes, added Terry Pizzie, head of commercial, Horizon Discovery.

Before releasing its next generation of bioproduction cell lines, Horizon will evaluate the impact of Amplycell’s technology on the expression capability of its parental GS-null CHO-K1 cell line. If the evaluation is successful, both parties anticipate a long-term relationship, with the BOOST procedure being applied as standard to future generations of cell lines.

Under the terms of the agreement, Horizon will make an upfront payment to AmplyCell. Upon establishing a positive impact on the CHO cell line, AmplyCell will then be entitled to receive success-based development and commercial milestone payments and Horizon will have the nonexclusive worldwide rights to sublicense these BOOSTed CHO-K1 cell lines to third parties. AmplyCell is also eligible to receive tiered royalties based on the net sales of these sublicensed lines.

“We are hopeful that a successful evaluation of AmplyCell’s BOOST technology will lead to incorporation of this methodology for all future bioprocessing cell lines released by Horizon, noted Pizzie.

Horizon is evaluating the impact of Amplycell’s technology on the expression capability of Horizon’s parental GS-null CHO-K1 cell line. [Horizon Discovery]

Molecular Diagnostics: Tempus, University of Chicago Medicine Partner on Personalized Breast Cancer Treatment

Tempus recently announced it is partnering with breast cancer specialists and research teams at the University of Chicago Medicine and Biological Sciences (UCM) to detect novel patterns that can predict how patients will respond to treatment. The collaboration is designed to improve and personalize treatment for breast cancer patients.

Under the agreement, Tempus has agreed to provide molecular sequencing and analysis for patients being treated for breast cancer at UCM, a research institute and healthcare provider. Through advanced bioinformatics and machine learning, Tempus plans to analyze data from about 1,000 breast cancer patients and generate additional genomic data for a subset of those patients.

That patient population is roughly equal to the more than 1,000 breast cancer patients whose data has been sequenced and annotated, with clinical information that includes treatment and outcomes data, as part of The Cancer Genome Atlas, the largest publicly available dataset. Tempus will work with the University of Chicago-based Genomic Data Commons, a next-generation platform designed to enable data access, analysis, and sharing for cancer research.

Over the past six months, Tempus has launched clinical collaborations with the University of Pennsylvania’s Abramson Cancer Center (focused on immunotherapies), University Hospitals Seidman Cancer Center (ovarian and triple negative breast cancer), University of Michigan (pancreatic cancer), and Duke University School of Medicine (brain cancer).

The company has also launched a collaboration with the Mayo Clinic, focused on patients participating in studies related to immunotherapy for lung cancer, melanoma, bladder cancer, breast cancer, and lymphoma, as well as endocrine therapy for advanced breast cancer.

In the collaboration with UCM, Tempus will work directly with Olufunmilayo (Funmi) Olopade, M.D., professor of medicine and human genetics and dean for global health at the University of Chicago. Dr. Olopade specializes in cancer-risk assessment and individualized treatment for the most aggressive forms of breast cancer.

“We are excited to partner with Tempus on this initiative and eager to support its efforts to build the largest clinically annotated molecular data set in breast cancer. This could improve clinical care and lead to novel research opportunities,” Dr. Olopade said in a statement.

Tempus operates its own CLIA-certified lab optimized for high-throughput clinical next-generation sequencing with a current capacity of more than 50,000 patients annually. All sequencing includes RNA-Seq capture and germline. Sequencing is completed within about two–three weeks of receiving patient samples.

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